Community Update: FDA decision on the approval of Scholar Rock's apitegromab
Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed approval of the Biologics License Application (BLA) for apitegromab in the treatment of people living with SMA.
The company reported that the FDA issued a Complete Response Letter (CRL) citing observations at a manufacturing facility, with no other approvability concerns noted.
Scholar Rock stated that remediation of the manufacturing issues is already underway and that the company plans to resubmit the BLA, anticipating the FDA will act expeditiously once the issues are fully resolved.
In Europe, the Marketing Authorisation Application (MAA) for apitegromab is under review by the European Medicines Agency, with a decision expected around mid-2026. If approved, launch in Europe is anticipated in the second half of 2026.